According to CNN, the US Food and Drug Administration (FDA) announced the approval of Hemgenix, the world's first gene therapy drug for hemophilia type B, on the 22nd local time. The Australian pharmaceutical manufacturer CSL Behring set the price of one dose at 3.5 million US dollars, making it the most expensive drug in the world. Hemgenix is a one-time gene therapy based on adeno-associated virus vector. It is used to treat adults with hemophilia type B by intravenous injection in a single dose. It is a hereditary bleeding disorder. About one in every 40000 people suffers from this disease, most of whom are men. Gene therapy for hemophilia has a history of more than 20 years. Dr. Peter Marks, director of FDA Biological Products Evaluation and Research Center, said in a statement that although the treatment of hemophilia has made progress, the methods of preventing and treating bleeding may have a negative impact on the quality of life of individuals. FDA approval provides a new treatment option for patients with hemophilia type B. The American Institute of Clinical and Economic Review is an independent non-profit research institution responsible for analyzing the value of prescription drugs and other medical products. The institution confirmed to CNN that Hemgenix is now the most expensive drug in the world. The FDA approved Hemgenix based on the safety and effectiveness evaluation of two studies conducted on about 60 adult males. One study showed that the level of blood clotting protein in people taking the drug increased, the demand for standard treatment decreased, and the bleeding problem decreased by 54%. (Outlook New Times)
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Source：http://digitalpaper.stdaily.com/http_www.kjrb.com/kjrb/html/2022-11/29/content_545177.htm?div=-1
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